Patient as partner

Empowered patients are redefining the healthcare environment on their own terms.

Throughout history, healthcare providers and the pharma industry generally made the decisions for the patients. This is no longer the case — just go and ask a patient.

“In today's dynamic and rapidly evolving healthcare environment, patients rightfully demand an active and informed role in managing their disease,” says Niko Andre, Head of Global Medical Affairs at Roche and a strong advocate for patient-centricity. “And we have a lot to learn from them.”

He is passionate and invigorated when contemplating the future of healthcare and the vast potential to transform lives of patients through truly transformational medicines and healthcare services. He clearly envisions a future that will be patient-powered, with the patient perspective firmly embedded in every aspect of pharmaceutical development and care delivery.

Integrating the patient voice into drug development and care delivery is not only part our responsibility to do right by patients and society, but it brings a more personalised approach to healthcare.

“To achieve this, we are developing holistic patient-partnering strategies that encourage listening to patient needs before embarking on the development and project journey,” says Niko. But it wasn’t always the case.

Physician knows best? Maybe ask a patient.

Early relationships between patients and patient organisations and Roche had a very different flavour to it than it does today. Irmi Gallmeier, Senior International Health Policy Leader at Roche, doesn’t mince words: “In the past the relationship was typically financial. It was primarily us providing contributions to patient organisations for their awareness campaigns and capacity building” she says. She describes these early relationships as patient engagement, but the type of engagement that doesn’t come close to a partnership. “It was felt there was little, if any, need to strategically include a strong patient input in our business processes.”

More and more, patients are in the decision making seat.

The patient voice wasn’t considered critical to drug development. “The old way of thinking was that patients did what their physicians instructed them to do, especially before the massive proliferation of online health resources and patient communities,” says Nina Ahluwalia, Head of Product Development Patient Insights at Roche, whose team works to gather and interpret patient input and acts as expert consultants to the clinician scientists.

“We felt we had a moderate idea of what was best for patients, because our teams contained medical experts and clinical trial experts. There was some thought about patients’ preferences, but limited opportunities to engage them directly and actually ask them about their experiences and preferences,” says Nina.

“It wasn’t really the norm to involve them in planning a clinical trial. If you think about it now from a patient’s perspective, and how much we ask of them emotionally, mentally and physically to participate in trials, and how central their participation is to our success, it’s rather obvious that they should have a say in our development,” she says.

The patient takes control

With increasing competition, the rise in personalised healthcare and an increasingly empowered (and vocal) patient population demanding trials and treatments that better fit their needs, Roche saw a need and a challenge. A group in Product Development (PD) came up with a positive solution. “The objective was to gather patient input and put their experiences, needs, and preferences at the center while planning trials,” Nina says.

Patients, caregivers and Roche at a patient organisation meeting in Muscat, Oman.

Moving from simple engagement with patient groups evolved into an increased level of patient partnership in the development process. This has led to a wake-up call in terms of focusing on patient-relevant value; value defined by patients. In the need for better clinical trial design, it is critical to focus on what patients deem successful, as opposed to Roche making assumptions about what “valuable” constitutes.

Currently there is no regulation from main health authorities, such as the FDA and EMA, that require proof of patient input in clinical trial design or drug development; there are only guidelines. “But if you discuss a valuable study goal with the FDA they will ask about patient input. From a regulatory perspective, you’ll need to bring proof of patient input, but how companies interpret them is up for debate” says Mathias Leddin, Data Scientist in pRED at Roche.

He stresses the importance of Roche including the patient perspective in clinical trial design, especially when it comes to benefiting the patient. “It’s essential to discuss endpoints with patients because it’s no longer about just survival, it’s about value and quality of life.”

It’s not as simple as asking: did the tumor shrink or not? When it comes to living with a disease, only patients can define what value means. There’s no way to know what a patient-relevant value is if we don’t ask them.

The future is a two way street

Irmi explains that “the new paradigm would be a true mutually beneficial patient partnership where we work together directly with patients and patient groups on a more systematic basis. This will bring their perspectives and insights to the entire continuum of our medicine lifecycle and involve them strategically in the major decisions we are making as a company.”

“It’s about developing medicines and assessing our medicines so that they have a patient-relevant value,” she adds.

Talking to individuals with ASD reveals surprises and insights

Early in the planning of a clinical trial, coming together in Patient Advisory Boards — an official meeting and information gathering session between Roche and patients or caregivers — is the moment for us to understand a patient’s journey, their motivations and barriers to participating in clinical trials. “The connection established during advisory boards between patients and the team members is powerful, and the mutual empathy we gain drives all of us to work together as partners,” says Nina. “Our clinical development teams walk away with a better sense of what patients like and don’t like about our trial, practical advice on how to improve upon their experience and more effectively educate patients about trial specifics like the design and purpose, and what to expect with procedures and tests. We are invariably surprised by some of their input; there’s always at least a few nuggets where we say ‘wow we didn’t realise they feel this way’”.

Patients have rightfully stepped up to become empowered owners of their decisions, their data and their future.
Niko Andre, Head of Medical Affairs

One such nugget came out of the Patient and Support Partner Advisory Board in anticipation of a clinical trial in autism. People with Autism Spectrum Disorder (ASD) do not refer to themselves as patients; they are “individuals with ASD” or “self-advocates”. “They’re high-functioning autistic adults; they’re not sick. They just communicate and interact with the world a little differently. It caught us by surprise and resulted in a real change in our approach. The team went back and rewrote communication material to reflect their perspective and respect their unique experience.”

The team also gathered that people with ASD don’t consider people who support them as caregivers in a traditional sense. High-functioning autistic individuals are assisted by friends and family, most of whom also don’t happen to meet the guideline definition of a caregiver. However, the primary endpoint for the planned trial, accepted by regulatory agencies, is completed by a caregiver or support partner - someone who is able to observe the individual with autism and who can complete assessments as part of the clinical trial. The team went back again and rewrote the clinical trial's definition of a caregiver, to better match the real-life relationships between people with ASD and their loved ones.

How to gather patient input: ask them

Donna Murray, Vice President of Clinical Programs & Head of Autism Treatment Network at Autism Speaks, an autism advocacy organisation based in the US, played a pivotal role at the Advisory Board between Roche and individuals with ASD. “It was refreshing to bring together self-advocates, families, clinicians, researchers and pharma to discuss the needs and challenges required to successfully do research with this population.”

Only patients can tell us what a meaningful outcome is.

“Clinical trials can be very demanding and it’s incredibly important that we work together with families and self-advocates to really find out what they want and need, and to identify the reservations and concerns towards participation in clinical trials,” she explains.

“Roche was sensitive to addressing the needs of this community — beyond the specific clinical trial as well. The information they gathered and applied proves how these interactions inform how we can work together to advance care for individuals with ASD more broadly; including how we can improve the ability to successfully run clinical trials for those with ASD.”

This partnership with patients was essential to the success in recruiting and retaining participants for the trial, but it also helps produce a more meaningful outcome. Patient partnership is still happening on an ad hoc basis and, according to Nina, that’s not enough. In order to establish the patient as a true partner, it has to become more ingrained in the entire product life cycle.

We can only bring the evidence of what is a patient relevant value if we work together with them, only then can we provide it and we can only do that together with patients as partners.
Irmi Gallmeier, Senior International Health Policy Leader

Niko recognises that “patient groups and Roche share a powerful joint vision: to improve patients’ access to innovative treatments. This is why we see patients as our partners, and are putting it into practice. We cannot develop successful medicines without the insights and involvement of patients, caregivers and patient groups.”

“Embedding the patient’s perspective and voice into everything we do has never been more important than today,” he adds.

Always a partner, right from the beginning

Patient engagement strategies are quickly being implemented into integral business practices to strengthen the efficiency of our medicines, and meet the needs of empowered patients. Nina describes how “the goal is based on the necessity to embed patient input in our processes. Patient input in clinical trial design must be a systematic and strategic pillar of how we do our business.”

Niko Andre, Head of Global Medical Affairs at Roche and a strong advocate for patient-centricity.

One example is the upcoming launch of the Patient Platform which will provide disease education, awareness of Roche clinical trials, and recruitment and clinical trial results directly to patients, making it easier for caregivers and patients to connect to possible clinical trial sites. “Responding to the public and regulatory demand, we have committed to provide lay person summaries with clinical trial participants within one year of study completion,” Niko explains.

Another measure is to roll out a systematic and strategic process to design clinical trials based on patient input across the molecule lifecycle, and to discuss the patient engagement strategy in drug development by disease area on a franchise level.

Nina reports that the first pilot was a moderate success. With some adjustments, the model will move to the next phase and be ready for implementation more widely through all of Roche’s franchises.

All of these activities are reviewed by patients before being implemented.

At the core of everything

In today’s fast-paced, complex and highly competitive business landscape, there’s no time to lose. Every medicine and treatment must be developed in partnership with patient groups in order to provide relevant and real value to patients and caregivers. And Niko points out what is at stake.

“The company is very much in a transformation mode and patient partnerships present incredible opportunities to evolve our business model to serve society better. This transformation — to innovate with patients — will require a huge mindset shift. Everyone in the company needs to be on board in order to adapt to the rapidly changing landscape,” he says. “The future of patient-centricity lies in a coordinated effort and a dedication to position the patient voice at the center of everything we do.

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